EP 3555294 A4 20200826 - REAGENTS FOR TREATMENT OF OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) AND USE THEREOF
Title (en)
REAGENTS FOR TREATMENT OF OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) AND USE THEREOF
Title (de)
REAGENZIEN ZUR BEHANDLUNG VON OKULOPHARYNGEALER MUSKELDYSTROPHIE (OPMD) UND VERWENDUNG DAVON
Title (fr)
RÉACTIFS POUR LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE OCULOPHARYNGÉE (DMOP) ET LEUR UTILISATION
Publication
Application
Priority
- US 201662434312 P 20161214
- AU 2017051385 W 20171214
Abstract (en)
[origin: WO2018107228A1] The present disclosure relates to RNA interference (RNAi) reagents, such as short hairpin microRNA (shmiR) and short hairpin RNA (shRNA), for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto. The present disclosure also relates to the use of the RNAi reagents in combination with PABPN1 replacment reagents, such as constructs which encode functional PABPN1 protein, for treatment of OPMD, compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.
IPC 8 full level
C12N 15/113 (2010.01); A61K 48/00 (2006.01); A61P 21/00 (2006.01)
CPC (source: CN EP IL KR RU US)
A61K 31/7088 (2013.01 - CN RU); A61K 48/00 (2013.01 - RU); A61K 48/005 (2013.01 - CN KR); A61P 21/00 (2018.01 - CN EP IL RU); A61P 27/02 (2018.01 - CN EP IL KR RU); C12N 15/113 (2013.01 - CN EP IL KR RU US); C12N 15/86 (2013.01 - CN KR); C12N 15/861 (2013.01 - RU); C12N 15/864 (2013.01 - RU); C12N 15/867 (2013.01 - RU); C12N 2310/14 (2013.01 - CN EP IL); C12N 2310/141 (2013.01 - CN IL KR US); C12N 2310/531 (2013.01 - CN EP IL KR US); C12N 2330/51 (2013.01 - CN EP IL KR US); C12N 2710/10043 (2013.01 - CN KR); C12N 2740/10043 (2013.01 - KR); C12N 2740/15043 (2013.01 - CN KR); C12N 2750/14143 (2013.01 - CN EP IL KR US); C12N 2800/22 (2013.01 - CN); C12N 2830/008 (2013.01 - CN KR)
Citation (search report)
- [A] MUHAMMAD RIAZ ET AL: "PABPN1-Dependent mRNA Processing Induces Muscle Wasting", PLOS GENETICS, vol. 12, no. 5, 6 May 2016 (2016-05-06), pages e1006031, XP055467448, DOI: 10.1371/journal.pgen.1006031
- [T] ALBERTO MALERBA ET AL: "Established PABPN1 intranuclear inclusions ino OPMD can be efficienly reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1", HUMAN MOLECULAR GENETICS, vol. 28, no. 19, 2 July 2019 (2019-07-02), pages 3301 - 3308, XP055703002, ISSN: 0964-6906, DOI: 10.1093/hmg/ddz167
- See also references of WO 2018107228A1
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
DOCDB simple family (publication)
WO 2018107228 A1 20180621; AU 2017377661 A1 20190718; AU 2017377661 B2 20230727; AU 2023222827 A1 20230914; BR 112019012312 A2 20200211; BR 112019012312 A8 20230228; BR 112019012312 A8 20230328; CA 3047154 A1 20180621; CN 110382698 A 20191025; CN 110382698 B 20240510; CN 118421624 A 20240802; EP 3555294 A1 20191023; EP 3555294 A4 20200826; IL 267353 A 20190829; IL 267353 B1 20230801; IL 267353 B2 20231201; IL 304674 A 20230901; IL 304674 B1 20240701; IL 304674 B2 20241101; JP 2020503014 A 20200130; JP 2023062092 A 20230502; JP 7233149 B2 20230306; JP 7431359 B2 20240214; KR 102495222 B1 20230209; KR 102667867 B1 20240529; KR 20190104151 A 20190906; KR 20230020586 A 20230210; MX 2019007110 A 20191205; MX 2024003643 A 20240415; RU 2019121950 A 20210118; RU 2019121950 A3 20210118; RU 2763319 C2 20211228; US 11142765 B2 20211012; US 11932852 B2 20240319; US 2019330629 A1 20191031; US 2022056446 A1 20220224; ZA 202106827 B 20220727
DOCDB simple family (application)
AU 2017051385 W 20171214; AU 2017377661 A 20171214; AU 2023222827 A 20230828; BR 112019012312 A 20171214; CA 3047154 A 20171214; CN 201780086338 A 20171214; CN 202410482087 A 20171214; EP 17882179 A 20171214; IL 26735319 A 20190613; IL 30467423 A 20230724; JP 2019532105 A 20171214; JP 2023023159 A 20230217; KR 20197019981 A 20171214; KR 20237003441 A 20171214; MX 2019007110 A 20171214; MX 2024003643 A 20190614; RU 2019121950 A 20171214; US 201716469992 A 20171214; US 202117463923 A 20210901; ZA 202106827 A 20210917